מחקר שלב 2 להרחבה בתווית פתוחה של AOC 1020 במשתתפים עם ניוון שרירים פציוסקפולוהומרלי (FSHD)
This study is an ongoing look at a new medicine called AOC 1020 for people living with Facioscapulohumeral Muscular Dystrophy (FSHD). If you were part of the previous AOC 1020 study, you might be able to join this one. The main goals are to keep checking how safe the medicine is, how well people tolerate it, and if it helps with FSHD over a longer period. Everyone in this new study will receive the AOC 1020 medicine, even if they had a dummy medicine (placebo) in the first study. The medicine will be given into a vein about every 6 to 7 weeks for roughly 4 years.
במבט חטוף
על מה המחקר הזה?
This study, called AOC 1020-CS2, is a follow-on to an earlier study for a medicine called AOC 1020. This medicine is being developed to help people with Facioscapulohumeral Muscular Dystrophy, often shortened to FSHD. FSHD is a condition that causes muscles to become weaker over time, especially in the face, shoulders, and upper arms, but can affect other parts of the body too.
The main purpose of this particular study is to continue making sure that AOC 1020 is safe to use over a longer period of time. It also aims to see how well people tolerate the medicine, meaning if they have side effects, and if it continues to show positive effects in managing FSHD. This kind of study, where everyone receives the active medicine, is important for gathering more long-term information after the initial tests.
By continuing to study AOC 1020, researchers hope to gain a much clearer picture of its potential benefits and any long-term effects. This information is a crucial step in understanding if AOC 1020 could become a helpful treatment option for people with FSHD in the future. The results from studies like this help doctors and regulators decide if a new medicine is safe and effective enough to be made widely available.
נקודות עיקריות
- This study is a follow-up for a medicine called AOC 1020 for FSHD.
- It aims to check the long-term safety and effects of AOC 1020.
- Only people from the previous AOC 1020 study can join.
- All participants will receive the active AOC 1020 medicine.
- The study involves regular medicine doses and check-ups over about four years.
- It's open to people aged 16 to 72.
מי עשוי להיות זכאי?
This study is specifically designed for people who have already finished the first AOC 1020 study. To be considered, your doctors in the first study must believe you tolerated the medicine well and followed the study rules properly. You must also be able to understand and sign consent forms, and be willing to stick to all the study appointments and rules.
There are also some reasons why you might not be able to join. For example, if you are pregnant, planning to get pregnant, or breastfeeding, you cannot join. You also need to be able to use effective contraception if needed throughout the study. If your doctor thinks you have any new or worsening health issues that would make it unsafe for you to take part, or that would make it difficult for you to complete the study, you would also not be eligible. This is to make sure the study is as safe as possible for everyone involved. Being unable to walk 10 meters without help specifically because of FSHD progression would not automatically stop you from joining.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- Have I completed the first AOC 1020 study?
- Did my doctors in that study think I tolerated the medicine well?
- Am I aged between 16 and 72 years old?
- Do I understand the need for careful contraception if applicable?
- Am I able to attend all the study visits for about four years?
- Am I currently pregnant, planning to be, or breastfeeding?
מה כרוך בהשתתפות?
If you join this study, you will receive the AOC 1020 medicine through a drip into your vein. This will happen roughly every 6 to 7 weeks. The active treatment part of the study will last for about 46 months (a bit under four years). After you finish receiving the medicine, you'll have an 18-week follow-up period where the study team will continue to check on your health and any side effects. In total, your involvement in the study could last up to about 50 months, which is just over four years. During this time, you will have regular visits to monitor your health, how you're feeling, and to ensure the medicine is being tolerated well. The study team will also keep track of any other medicines you take and, for women, your pregnancy status.
סיכונים ותועלות פוטנציאליים
מיקומים (17)
- University of California Los Angelesמיקוד מאומתלוס אנג'לס, ארצות הברית
- אוניברסיטת סטנפורדמיקוד מאומתפאלו אלטו, ארצות הברית
- אוניברסיטת קליפורניה בסן דייגומיקוד מאומתסן דייגו, ארצות הברית
- University of Coloradoמיקוד מאומתדנוור, ארצות הברית
- University of Floridaמיקוד מאומתגיינסוויל, ארצות הברית
- מחקר מחלות נדירותמיקוד מאומתאטלנטה, ארצות הברית
- Kansas University Medical Centerמיקוד מאומתקנזס סיטי, ארצות הברית
- University of Rochester Medical Centerמיקוד מאומתרוצ'סטר, ארצות הברית
- אוניברסיטת דיוקמיקוד מאומתדרהאם, ארצות הברית
- Ohio State Universityמיקוד מאומתקולומבוס, ארצות הברית
- University of Pennsylvaniaמיקוד מאומתפילדלפיה, ארצות הברית
- University of Texas Southwesternמיקוד מאומתדאלאס, ארצות הברית
שאלות נפוצות
What is FSHD?
FSHD stands for Facioscapulohumeral Muscular Dystrophy, a condition that causes muscles in the face, shoulders, and arms to weaken over time.
What is AOC 1020?
AOC 1020 is the name of the new medicine being tested in this study for people with FSHD.
מי יכול להצטרף למחקר זה?
This study is for people aged 16 to 72 who have already finished a previous AOC 1020 study and were found suitable by the study doctors.
כמה זמן אהיה במחקר?
The study could last up to about 50 months (just over four years), including the time you receive the medicine and a follow-up period.
Will I get the actual medicine or a dummy medicine?
Everyone who joins this specific follow-on study will receive the active AOC 1020 medicine.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
דיון
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